Safety & Efficacy Study of ORGN001 (Formerly ALXN1101) in Pediatric Patients With MoCD Type A Currently Treated With rcPMP
NCT ID: NCT02047461
Last Updated: 2023-10-17
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE2
8 participants
INTERVENTIONAL
2014-04-30
2022-10-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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ORGN001 (formerly ALXN1101)
daily IV infusions
ORGN001 (formerly ALXN1101)
IV infusion
Interventions
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ORGN001 (formerly ALXN1101)
IV infusion
Eligibility Criteria
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Inclusion Criteria
* Currently treated with rcPMP infusions
Exclusion Criteria
ALL
No
Sponsors
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Origin Biosciences
INDUSTRY
Responsible Party
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Locations
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Children's Hospital of Wisconsin
Milwaukee, Wisconsin, United States
Monash Medical Centre
Melbourne, , Australia
Beatrix Children's Hospital
Groningen, , Netherlands
Unité des maladies métaboliques
Tunis, , Tunisia
Royal Hospital for Sick Children
Glasgow, , United Kingdom
Manchester University Hospitals NHS Foundation Trust
Manchester, , United Kingdom
Countries
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Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Related Links
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FDA Drug Approval Package
Other Identifiers
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2013-002701-56
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
ALXN1101-MCD-201
Identifier Type: -
Identifier Source: org_study_id
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